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Targeting Genetic Modifiers of HBG Gene Expression in Sickle Cell Disease: The miRNA Option

12 May 2022

Abstract excerpt

Sickle cell disease (SCD) is one of the most common inherited hemoglobinopathy disorders that affects millions of people worldwide. Reactivation of HBG (HBG1, HBG2) gene expression and induction of fetal hemoglobin (HbF) is an important therapeutic strategy for ameliorating the clinical symptoms and severity of SCD. Hydroxyurea is the only US FDA-approved drug with proven efficacy to induce HbF in SCD patients,...

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Share to Epigenetics and DNA Methylation
Share to Hemoglobinopathies and Related Disorders
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PMID: 35553407
DOI: 10.1007/s40291-022-00589-z

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